Scientists behind a world-first gene therapy reveal how they teamed up with experts across academia and industry to produce a ...

In the first part of this series, we explored how early genetic screening and gene therapy transform the lives of newborns ...

UniQure said it has aligned with the FDA on components of a statistical analysis plan for a registrational trial of AMT-130.

In comments posted in response to the Trump administration’s pharma tariff investigation, companies and industry groups ...

While vepdegestrant outperformed a standard treatment option in a subtype of patients, the benefit on progression-free ...

As newborn screening and rapid DNA sequencing become routine, we are poised to catch and treat inherited diseases at their ...

STEMCELL Technologies, Canada’s largest biotechnology company, is pleased to announce the acquisition of Cellular Highways ...

AMT-130 has been granted the FDA’s Regenerative Medicine Advance Therapy (RMAT) designation and Breakthrough Therapy designation, the first therapy for Huntington’s disease to receive an RMAT ...

Greater numbers of circulating stem cells increase the efficacy of in vivo gene therapy techniques, potentially reducing the price tag of lifesaving treatments.

This comes at a challenging time for gene therapies, as multiple serious AEs and fatalities have been recently reported.

Aligning with EU on genetically modified food and agricultural products will create a regulatory cliffedge for a sector which ...

Intellia has leveraged its expertise in Crispr/Cas9 gene editing to advance a pipeline of in vivo and ex vivo therapies for diseases with high unmet medical needs. We believe Intellia's proprietary ...